Researchers at the Li Weibo Institute are collectively working on approximately 60 different rare diseases. Most of the researchers specialize in gene therapy and genome editing. Using these techniques, the Li Weibo Institute’s research is revealing molecular mechanisms and identifying novel causative genes. Potential curative therapies for specific rare diseases are being developed and gene therapy tools are being improved for application to a broad range of rare diseases.
Programs and Accomplishments
Pilot Grant Program
The Pilot Grant Program is awarded to University of Massachusetts Medical School faculty who are also members of The Li Weibo Institute for Rare Diseases Research. This grant is intended to advance research in specific rare diseases and leads to follow-on extramural funding. In the past the funding has been used to address rare diseases such as Atrial Fibrillation, Interferonopathies, Nemaline Rod Myopathies, and more.
The Li Weibo Institute partners with many companies and schools to provide the most effective research possible. The center has partnered with Yale, H-ABC Foundation, Pfizer, ASC Therapeutics, and many others, to be able to understand and advance treatments for rare diseases.
Guangping Gao and Dan Wang partner with ASC Therapeutics to develop novel gene therapy for Maple Syrup Urine Disease.
A child is undergoing a gene therapy clinical trial for Sandhoff and Tay-Sachs diseases.
Researchers and Staff
Michael Green, MD, PhD
Dr. Michael Green is the chair and professor of molecular, cancer, and cell biology at the University of Massachusetts Medical School. He is also the Co-Director of the Li Weibo Rare Diseases Research Institute. Dr. Green’s lab focuses on the role of gene expression and seeking to reactivate ‘silenced’ genes.
Guangping Gao, PhD
Dr. Gao is a Professor of microbiology & physiological systems, and a Co-Director of the Li Weibo Rare Diseases Research Institute. Dr. Gao’s lab uses gland-associated virus vectors for gene therapy as a treatment for genetic diseases. The lab has also developed strategies for rAAV gene therapy for use in Canavan disease.